In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

In recent years recombinant adeno-associated viral vectors (AAV) have become
increasingly valuable for in vivo studies in animals, and are also currently being tested in …

Gene delivery using recombinant adeno-associated virus (rAAV) has emerged to the
forefront demonstrating safe and effective phenotypic correction of diverse diseases …

Recombinant adeno-associated virus vectors based on serotype 2 (rAAV2) can direct
transgene expression in the central nervous system (CNS), but it is not known how other …

Adeno-associated virus (AAV) vectors often undergo long-distance axonal transport after
brain injection. This leads to transduction of brain regions distal to the injection site, although …

Therapeutic gene delivery to the whole spinal cord is a major challenge for the treatment of
motor neuron (MN) diseases. Systemic administration of viral gene vectors would provide an …

The potential host immune response to a nonself protein poses a fundamental challenge for
gene therapies targeting recessive diseases. We demonstrate in both dogs and nonhuman …

Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to
administer a therapeutic transgene from the innervated musculature. With the aim of scaling …

Adeno-associated virus (AAV) gene therapy constitutes a powerful tool for the treatment of
neurodegenerative diseases. While AAVs are generally administered systemically to …

Various regions of the brain have been successfully transduced by recombinant adeno‐
associated virus (rAAV) vectors with no detected toxicity. When using the cytomegalovirus …